Lysogene announced it has entered into a manufacturing agreement with viral gene and cell therapy manufacturer Brammer Bio.
Brammer Bio will produce LYS-GM101, an AAVrh10-based gene therapy, for clinical testing of the therapeutic candidate in patients with GM1 Gangliosidosis, a rare neuronopathic lysosomal storage disorder.
“We are pleased to have secured Brammer Bio, a leading manufacturer with proven expertise in the development of robust industrial-scale manufacturing of AAV-based products, to provide the highest quality LYS-GM101 product for clinical testing,” Mark Plavsic, Chief Technical Officer at Lysogene said. “This agreement ensures Lysogene has established commercial-ready gene therapy manufacturing in line with our need for the product.”
Pre-clinical data in animal models of GM1 show that LYS-GM101 treatment delivers a functional gene encoding the βgal enzyme resulting in a reduction of GM1 gangliosides and transforms the animal phenotype. These studies will support an Investigational New Drug application and the launch of the Phase I clinical trial, expected to launch in 2019.
"We are delighted to partner with Lysogene to manufacture this commercial-ready AAVrh10 gene therapy product,” Mark Bamforth, President and CEO of Brammer Bio said. “We embrace this opportunity to help patients in need and re-affirm Brammer Bio’s leadership position as the manufacturer of choice in the gene therapy space."
GM1 is an extremely severe, autosomal recessive disease caused by a mutation in the GLB1 gene encoding for the lysosomal acid beta-balactosidase (ßgal) enzyme. The resulting enzymatic deficiency leads to accumulation of GM1-ganglioside in cells. Clinical presentation is mainly neurological with rapidly progressive impairment (motor, cognitive and behavioral) leading to premature death, mostly in early childhood. It is a devastating disease for patients and families. There is currently no disease modifying treatment available.